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Experimental Drug Being Tested in Mitochondrial and Metabolic Disorders, FA

Update (Feb. 13, 2013):In February 2013, study coordinator Lynne Wolfe at the National Institutes of Health said the NIH study of EPI-743 in metabolic and mitochondrial disordersis seeking children with undiagnosed, suspected disorders of energy metabolism. Children who have a gentically confirmed metabolic or mitochondrial disorder will not be accepted into this study.

EPI-743 is a small-molecule, oral drug that is designed to enter the central nervous system and synchronize (improve) energy generation in mitochondria, the energy-producing subunits in cells. It is being developed by Edison, which is located in Mountain View, Calif., and Utrecht, Netherlands.

FA trial planned, access program closed

Edison also is planning a trial to test the effect of EPI-743 on visual function in people with FA. Study sites are likely to include the University of South Florida in Tampa, Children's Hospital of Philadelphia, and the University of California, Los Angeles.

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.